Advancing Gene Therapy: KJ’s Story

After nearly 40 years at Children’s Hospital of Philadelphia (CHOP), including 11 as CEO, Madeline Bell will retire in October. In the final episode of Breaking Through, Madeline introduces CHOP President Dr. Joe Mitchell, who will succeed her as CEO, and reflects on her time at CHOP.

For Philadelphia Eagles President and longtime Children’s Hospital of Philadelphia (CHOP) volunteer Don Smolenski, success is built on trust and care – and connecting with the community means everything. In the new episode of Breaking Through, Don joins Madeline to talk about what the team means to the city – and what makes the partnership between the Eagles and CHOP so special. It’s an inspiring conversation that highlights how a collaboration built on shared values can make a difference for an entire community.  

When baby KJ was diagnosed with a rare metabolic disorder, the team at Children’s Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ’s first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients. 

Earlier this year, baby KJ became the first patient in the world to receive a personalized gene editing therapy, which was developed by a team at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine. In June, KJ went home after spending more than 300 days at CHOP. On this episode of Breaking Through, Madeline speaks to KJ’s parents, Kyle and Nicole, about the treatment that changed KJ’s life – and how he’s doing today.

This Women’s History Month, we are celebrating the life and legacy of one of Children’s Hospital of Philadelphia’s original breakthrough makers: pediatric oncologist Dr. Audrey Evans. In 1969, Dr. Evans became the first woman to serve as Chief of CHOP’s Division of Oncology, and she went on to co-found the first Ronald McDonald House. AUDREY’S CHILDREN, a film based on Dr. Evans’ life, will be released in theaters across the United States on March 28. In this episode of Breaking Through, Julia Fisher Farbman, the film’s producer and writer, joins Madeline to discuss Dr. Evans’ extraordinary life and career.

Teams at Children's Hospital of Philadelphia (CHOP) are committed to delivering breakthroughs for patients, including keeping them safe from injury – the leading cause of death and acquired disability for children. In this episode of Breaking Through, Madeline talks to CHOP expert Dr. Kristy Arbogast about how caregivers can keep kids safe during fall sports season and beyond – and about the work CHOP’s Center for Injury Research and Prevention is leading to find new ways to prevent and treat concussions. They also discuss Dr. Arbogast’s role on the NFL Engineering Committee, which is focused on developing injury prevention programs for elite athletes.

Sickle cell disease is an inherited blood disorder that often causes debilitating pain and life-threatening complications. Many patients with the disease make dozens of ER visits a year, and must receive regular blood transfusions. And though recent breakthroughs in gene therapy are giving patients with sickle cell new hope – and new options – accessing these therapies can be challenging. In this episode of Breaking Through, cell and gene therapy expert Dr. Stephan Grupp and pediatric hematologist Dr. Alexis Thompson join Madeline to talk about how gene therapy for sickle cell disease can transform patients’ lives. They also discuss what teams at Children’s Hospital of Philadelphia (CHOP) are doing to ensure that these innovative therapies reach the children who need them most. 

The team at Children’s Hospital of Philadelphia (CHOP) believes that mental health is just as important as physical health – and they’re working together to find solutions to the pediatric mental health crisis. In this episode of Breaking Through, Dr. Catharyn Turner, CHOP’s Medical Director of Acute Inpatient Psychiatry, joins Madeline to discuss the innovative work CHOP is leading to improve access to pediatric mental health care, CHOP's new mental health facilities in West Philadelphia, and what’s behind the rise in mental health conditions in children and teens.

From the time Elijah was a baby, he had many painful symptoms – and no one could determine what was wrong. But when Elijah was a toddler, his family finally found answers at Children’s Hospital of Philadelphia. In this episode of The Breakthrough Chronicles, 12-year-old Elijah and his parents talk to Madeline about how teams at CHOP transformed his health – and his life.

Tenlee was born with a rare heart condition and had a heart transplant at Children’s Hospital of Philadelphia when she was just 5 months old. And though she’s doing well today, her journey has been very challenging. In this episode of The Breakthrough Chronicles, Tenlee’s parents, Jami and Nick, speak with Madeline about the challenges Tenlee has overcome – and how their family’s experience at CHOP changed their lives.